Author(s): Shi Q, Montgomery RR
Abstract Share this page
Abstract Platelets are small, anucleate, discoid shaped blood cells that play a fundamental role in hemostasis. Platelets contain a large number of biologically active molecules within cytoplasmic granules that are critical to normal platelet function. Because platelets circulate in blood through out the body, release biological molecules and mediators on demand and participate in hemostasis as well as many other pathophysiologic processes, targeting expression of proteins of interest to platelets and utilizing platelets as delivery systems for disease treatment would be a logical approach. This paper reviews the genetic therapy for inherited bleeding disorders utilizing platelets as delivery system, with a particular focus on platelet-derived FVIII for hemophilia A treatment. Copyright © 2010 Elsevier B.V. All rights reserved.
This article was published in Adv Drug Deliv Rev
and referenced in Journal of Genetic Syndromes & Gene Therapy